Experimental Therapeutics in Pediatric Cancer

Nationally ranked by U.S. News & World Report

Experimental Therapeutics in Pediatric Cancer

Nationally ranked by U.S. News & World Report

The Experimental Therapeutics in Pediatric Cancer program makes clinical trials available to your child when traditional cancer therapies have not been successful.

We are the only pediatric hospital in the region that provides access to cancer clinical trials, which means your child can benefit from the latest innovations in research while staying close to home. This is important because family connection and familiarity with surroundings has been shown to improve the success of cancer treatment.

What is experimental therapeutics?

Our trials test treatment methods that have not yet been used for your child’s diagnosis. Many medications involved in clinical trials have been proven effective and safe for other cancers or for use in adult patients, but not specifically for pediatric cancer. Experimental Therapeutics is the science of taking those treatments and trialing them with different cancers to test their effectiveness.

What are the phases of a cancer research trial?

Phase 1: A test of a drug to determine its safety and proper dosage for pediatric patients. At this phase, we do not know yet if a drug is effective.

Phase 2: This phase comes after a drug’s safety has been proven and tests the drug’s effectiveness.

Phase 3: This phase ensures a drug meets our standard of care. Phase 3 is designed to verify if a drug can improve on how we currently treat a diagnosis.

Financial assistance to help fund your trial participation

If your child qualifies for a clinical trial in our program, we provide guidance in finding financial resources to help cover the cost of the trial. In addition to working with Missouri and Kansas Medicaid programs, we also assist with access to Medicaid programs in other states. Many parts of the clinical trial’s process, such as labs, X-rays, and other essentials tests, are covered by your health insurance.

The research team

The Experimental Therapeutics Research Team works to provide the best in cancer research expertise while providing the emotional support your family needs. Our team includes four doctors, one nurse practitioner, four research coordinators, and one social worker.

Investigational Drug Service (IDS) Pharmacy

The Children’s Mercy IDS Pharmacy is a full service pharmacy providing clinical knowledge and operational support for all studies involving investigational drugs at Children’s Mercy. As part of the Experimental Therapeutics Team, the IDS Pharmacy staff provides protocol and sponsor directed security, accountability, education, and dispensing of all Investigational as well as ancillary medications.

Cancer trials currently available

This list changes frequently and does not include most of our Children’s Oncology Group trials.

AALL1521: A Phase 2 Study of the JAK1/JAK2 Inhibitor Ruxolitinib With Chemotherapy in Children With De Novo High-Risk CRLF2-Rearranged and/or JAK Pathway–Mutant Acute Lymphoblastic Leukemia

Refractory Solid Tumors

ADVL1322: A Phase 2 Study of Pazopanib in Children, Adolescents, and Young Adults with Refractory Solid Tumors

Relapsed/Refractory Juvenile Myelomonocytic Leukemia

ADVL1521: Phase 2 Study of the MEK inhibitor Trametinib in Children with Relapsed or Refractory Juvenile Myelomonocytic Leukemia

Refractory Solid Tumors

ADVL1622: A Phase 2 Trial of XL184 (Cabozantinib) an Oral Small-Molecule Inhibitor of Multiple Kinases in Children and Young Adults with Refractory Sarcomas, Wilms Tumor, and Other Rare Tumors

Relapsed/Refractory Classic Hodgkin Lymphoma

AHOD1721: A Phase 2 trial of nivolumab + brentuximab vedotin for children, adolescents, and young adults with relapsed/refractory CD30 + classic Hodgkin lymphoma after failure of first-line therapy, followed by brentuximab +bendamustine for participants with a suboptimal response.

Anaplastic Large Cell Lymphoma

ANHL12P1: A Randomized Phase 2 Trial of Brentuximab Vedotin or Crizotinib in Combination with Chemotherapy for Newly Diagnosed Patients with Anaplastic Large Cell Lymphoma

CDRB436G2201: Phase 2 Open-Label Global Study to Evaluate the Effect of Dabrafenib in Combination with Trametinib in Children and Adolescent Patients with BRAF V600 Mutation Positive Relapsed or Refractory High Grade Glioma (HGG)

Relapsed/Refractory Neuroblastoma or Medulloblastoma

V0706: A Phase 2 Trial of Nifurtimox for Refractory or Relapsed Neuroblastoma or Medulloblastoma

AflacST1502: A Phase 2 Study of Sirolimus in Combination with Metronomic Chemotherapy in Children with Recurrent and/or Refractory Solid and CNS Tumors

Relapsed/Refractory Solid Tumors

I5B-MC-JGDN: A Phase 1, Open-Label, Dose-Escalation Study of Olaratumab as a Single Agent and in Combination with Doxorubicin, Vincristine/Irinotecan, or High-Dose Ifosfamide in Pediatric Patients with Relapsed or Refractory Solid Tumors

Children's Research Institute

The Children’s Research Institute at Children's Mercy is creating an integrated research environment where no boundaries exist between science and medicine. In our quest to find answers to pediatric medicine’s most challenging questions, we are collaborating with physicians, scientists, academic colleagues, philanthropic partners and others within our community, and around the world.